“I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”
“I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”
ABI-201 is an engineered AAV.N54 vector that expresses a soluble CD59 variant and a complement 3 (C3) inhibitor fusion protein. ABI-201 was developed by Avirmax Biopharma Inc. to confer photoreceptor protection against retinal damage. The effects of ABI-201 were tested in a sodium iodate-induced retinal damage model in nonhuman primates, a widely used model for testing therapies for age-related macular degeneration (AMD).
Viverita Therapeutics Inc. has established a strategic research collaboration with Boehringer Ingelheim Pharma GmbH & Co. KG aimed at accelerating the discovery and validation of novel therapeutic targets for cancer.
Interleukin-10 (IL-10) is an immunoregulatory cytokine produced by a variety of immune cells, including type 1 regulatory T cells and macrophages, as well as nonimmune cells such as epithelial cells and fibroblasts.
Genprex Inc. and University of Pittsburgh have entered into a new sponsored research agreement (SRA) to study GPX-002, Genprex’s gene therapy for diabetes in animal models of type 1 and type 2 diabetes.
Robert Williamson, CEO of Triumvira Immunologics, and Hernan Bazan, CEO of South Rampart Pharma, discuss bracing for tariffs, NIH grant cuts and gifting China a biopharma leadership position.
Cadherin 17 (CDH17) is a membrane-bound cell adhesion molecule involved in tumor cell proliferation and is selectively overexpressed in several gastrointestinal malignancies, including colorectal cancer, gastric cancer and pancreatic cancer.
Recent evidence has suggested that the use of norrin mimetics targeting both frizzled-4 (FZD4) and low-density lipoprotein receptor-related protein 5 (LRP5) may be highly effective at modulating retinal vascular leakage. When combined with an anti-VEGF therapy, it was hypothesized to have an...
Glaucoma is a leading cause of blindness and is characterized by the death of retinal ganglion cells (RGCs). Genetic screening data have identified dual leucine zipper kinase (DLK) and leucine zipper kinase (LZK) as important mediators of RGC death.
Formosa Pharmaceuticals Inc. and Almac Discovery Ltd. have announced a global licensing agreement for development and commercialization of ALM-401, a first-in-class engineered bispecific antibody-drug conjugate (ADC) for the treatment of solid tumors characterized by the dual expression of EGFR and...
Researchers from the Access to Advanced Health Institute and collaborating institutions have developed a promising new bivalent vaccine candidate that can protect against both yellow fever and Zika viruses. The study demonstrates that the vaccine elicits robust humoral and cellular immune responses...
Scientists at Institut Pasteur have gained new insights into how some people control HIV-1 replication after interruption of antiretroviral treatment (ART). The investigators found a fingerprint involved in long-term viral remission.
Armatus Bio Inc.’s development of ARM-201, an AAV-delivered microRNA therapy for facioscapulohumeral muscular dystrophy (FSHD), has been boosted by a $3 million investment by Solve FSHD.
Alchemab Therapeutics Ltd. has entered into a licensing agreement with Eli Lilly and Co. for ATLX-1282, Alchemab’s first-in-class IND-ready program targeting a novel receptor and mechanism for amyotrophic lateral sclerosis (ALS) and other neurodegenerative conditions.
Abnormal aggregation of α-synuclein is thought to contribute to the pathology of Parkinson’s disease, the second most frequent neurodegenerative disorder. Therefore, developing drugs that prevent such aggregation could be effective for slowing or even preventing the disease.
Unravel Biosciences Inc. has submitted clinical study applications in Colombia seeking to begin proof-of-concept clinical trials for RVL-001 for Rett syndrome and Pitt-Hopkins syndrome.
Mammoth Biosciences Inc. has nominated its first clinical development candidate – MB-111 – a potential one-time treatment for patients with very high triglycerides, including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia. IND-enabling studies are on track to begin this year.
The Mediator complex is a system that regulates protein-coding gene transcription, where mediator of RNA polymerase II transcription subunit 16 (MED16) is a subunit belonging to this system. Pathogenic genetic variants in Mediator complex subunits usually lead to neurodevelopmental and...
Investigators from the University of Pennsylvania have presented data regarding the relationship of angiopoietin-2 (ANG2) and its prognostic impact on traumatic brain injury (TBI). The ANG2 plasmatic levels were measured in patients with TBI (n=362), orthopedic injury controls (n=89) and healthy...
Ignite Biomedical Inc. and Liquid Biosciences have announced the discovery of biomarkers to diagnose substance use disorder (SUD). The set of biomarkers includes some that have not previously been associated with addiction and spans all 6 SUDs (opioid, cocaine, methamphetamine, alcohol, nicotine...
Equillium Inc. is advancing EQ-504, a potent aryl hydrocarbon receptor (AhR) modulator acquired through the acquisition of Ariagen Inc., a company that was majority owned by Equillium’s largest investor, Decheng Capital.
Bristol Myers Squibb Co. has synthesized receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of inflammatory bowel disease, psoriasis, rheumatoid arthritis, nonalcoholic or...
Parvus Therapeutics Inc. has announced the achievement of a milestone under its collaboration with Abbvie Inc. to develop treatments for inflammatory bowel disease (IBD). PVT-401, a novel peptide-major histocompatibility complex (pMHC)...
A large-scale study has revealed the impact of germline variants on proteins in 10 cancer types. Scientists from the National Cancer Institute’s Clinical Proteomic Tumor Analysis Consortium (CPTAC) conducted a precision proteogenomic analysis in a...
Breakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.